The Australian Government listed nusinersen on the Pharmaceutical Benefits Scheme (PBS) from 1 June 2018 for the treatment of Type 1, Type 2 and Type 3a spinal muscular atrophy (SMA)

Page last updated: 02 January 2019

Nusinersen (trade name Spinraza) is marketed by the pharmaceutical sponsor Biogen Australia Pty Ltd (Biogen) as a treatment for SMA, a rare genetic neuromuscular disorder.

Biogen submitted a request to subsidise nusinersen through the Australian Government’s PBS for the treatment of Type 1, Type 2, and Type 3a SMA. It is expected that around 160 patients will receive treatment every year as a result of this listing.

The PBAC, an independent, expert group, makes recommendations to the Australian Government about PBS listings.

In March 2018, the PBAC recommended the Section 100 (Highly Specialised Drugs Program) listing of nusinersen for the treatment of paediatric patients with infantile-onset or childhood onset SMA with onset of symptoms prior to three years of age.

Patients would otherwise pay $367,850 a year for this medicine. The PBS listing will reduce the cost for patients to a maximum of $39.50 per script for general patients, and $6.40 for concessional patients.

The PBAC was satisfied that based on the clinical evidence available, nusinersen provides a significant improvement in efficacy over standard of care for the proposed patient population.

In July 2018, the PBAC recommended the current listing for nusinersen be extended to include Section 100 (Highly Specialised Drugs Program) Private Hospitals.

The PBAC’s original decision can be found on the PBS website.

The PBAC’s view on the change to include access through Private Hospitals can be found on the PBS website.

The PBS listing of nusinersen was extended to Private Hospitals on 1 November 2018.